Effect of Training on Brain Volume in Ataxia
Spinocerebellar AtaxiasThe primary aim is to show balance training improves DCD individual's ability to compensate for their activity limitations, but does not impact disease progression. The second aim is to demonstrate aerobic exercise improves balance and gait in DCD persons by affecting brain processes and slowing cerebellar atrophy.
Clinical Trial With Riluzole in Spinocerebellar Ataxia Type 2 (ATRIL)
Spinocerebellar Ataxia Type 2ATRIL is a multi-centric, double-blind randomized, two-arm controlled study. 42 SpinoCerebellar Ataxia type 2 (SCA2) patients, both gender, at least 18 years of age will be included. Riluzole 50 mg will be administered (per os) twice a day, versus one group with placebo for 12 months. Riluzole (Rilutek®) is a benzothiazole drug, market approved, for Amyotrophic Lateral Sclerosis (ALS). It delays the onset of ventilator-dependence or tracheostomy in selected patients and may increase survival. Scale for the Assessment and Rating of Ataxia (SARA) will be used at M0, M6 and M12. To assess primary criterion, the percentage of patients with a decrease of at least 1 point of the SARA score between the inclusion visit, and Visit 3 (Months 12) will be calculated.
Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich...
Friedreich AtaxiaThe purpose of this study is to evaluate the efficacy of TAK-831 versus placebo on upper extremity (arm and hands) motor function and manual dexterity. This study will also evaluate the efficacy of TAK-831 versus placebo on activities of daily living (ADL) and other secondary assessments.
Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia
Friedreich AtaxiaThe purpose of this study is to evaluate the safety, tolerability, pharmacokinetic and pharmacodynamic effects of intravenous DT-216 in adult patients with Friedreich Ataxia. This single ascending dose study is randomized, double-blind, placebo-controlled.
A First in Human Study of RT001 in Patients With Friedreich's Ataxia
Friedreich's AtaxiaThe purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of RT001 in patients with Friedreich's ataxia.
Methylprednisolone Treatment of Friedreich Ataxia
Friedreich AtaxiaThis study will explore whether methylprednisolone treatment is safe, well-tolerated, and beneficial in patients that are diagnosed with Friedreich Ataxia (FRDA). The study will also explore if methylprednisolone has any effects on biomarkers associated with FRDA. All subjects in the study will receive the same steroid treatment.
Transcranial Magnetic Stimulation in Spino-Cerebellar Ataxia
Spinocerebellar AtaxiaSpinocerebellar Ataxia (SCA) refers to a family of genetic diseases that cause progressive problems with gait and balance, as well as other debilitating symptoms. This is a randomized controlled pilot study to test a novel therapeutic intervention that uses noninvasive magnetic brain stimulation to improve functional outcomes in patients with SCA. The study will include quantitative evaluations of gait, balance, and brain physiology to examine possible objective end-points for a future, larger multi-site clinical trial. The investigators anticipate that patients receiving the real intervention will show a functional gain.
(+) Epicatechin to Treat Friedreich's Ataxia
Friedreich's AtaxiaThis 24-week study will test the safety and effectiveness of synthetically produced (+) Epicatechin in treating patients who have Friedreich's Ataxia, a neurological disorder.
Citocoline for Treatment in Fragile X-associated Tremor/Ataxia Syndrome
Fragile X Tremor/Ataxia SyndromeThe objective of this study is to determine if citocoline is effective for balance abnormalities and to stabilize cognitive decline in patients with fragile X-associated tremor ataxia syndrome. The study will test 1000mg twice daily of citocoline for 12 months in an open label pilot study, with study visits at baseline, 3, 6, and 12 months.
Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia...
Friedreich's AtaxiaThis study is meant to assess the safety and tolerability of idebenone in patients with Friedreich's Ataxia over a 12 months period.